从以学统计学看大规模临床试验的概念-张学中选编.ppt

从医学统计学看大型临床试验的概念;Ⅰ.已进入随机的病人可以退出吗？;Ⅰ.已进入随机的病人,研究人员可以使其出组吗？ 答：不。病人的排除标准仅在随机分组时适用。病人一旦被纳入研究（随机分为各研究性治疗组）， 则不论其是否接受了研究药物治疗，均不从研究中做出组处理。全部病人的7天和30天的随访表必须填齐。;Can a patient be withdrawn from the study once randomized? No. The patient exclusion criteria apply only at the time of randomization. Once a patient is entered (randomized to study treatments), the patient cannot be withdrawn from the study regardless of whether patient received study treatments or not. The 7 and 30-Day Follow-up Forms must be completed for all patients. ;Can a participant leave a clinical trial after it has begun?Yes. A participant can leave a clinical trial, at any time. When withdrawing from the trial, the participant should let the research team know about it, and the reasons for leaving the study. ;临床试验中的两个原则;高血压临床试验中的四个概念;;;; ITT Principle; ITT 原则;Clin. Drug Invest.16(6),413-420,1998;Circulation.102(24),2940,December 12 ,2000;NEJM,345(8),575,Aug. 23,2001;Hill’s example 1 on ITT;随机临床试验方案;随机临床试验 结果;出组 withdrawal 离组 Drop out 错组 Drop in; RCT的分析方法;Two-year mortality in the coronary artery bypass surgery trial published by The European Coronary Surgery Study Group (1979)(upper part) and results of the analysis carried out according to the intention-to-principle and in terms of clinical efficacy;; 对ITT尖锐的批评;;全分析集;符合方案集; 研究总体 研究人群,研究样本 目标总体;研究人群 study population;研究总体 study population;目标总体 target population;ITT人群 intent to treat population;目标总体和研究总体;研究总体和研究样本;再谈ITT原则; 治疗意向原则（1）; 贯彻处理意向原则（2）; ITT原则，另外的说法; ITT原则的沿革;RISK in 2003ESHESC JNC7express;绝对危险,ar相对危险,rr优势比,比数比,or风险比,hr规因危险(特异危险),ar人群规因危险,Par;?? ?? ;?? ?? ;2003ESH/ESC定义的概念;Definition of risks; 1988年Laupacis等人提出了NNT的概念，??其用于疗效或危险的评价。 NNT被定义为绝对危险减少值的倒数，其含义是，在一段时间内如果1例事件发生，需要有经过治疗(观察)的病人数目。 NNT=1/(IE – IE) ,may be RR1=RR2,NNT1=2NNT2;绝对危险, 相对危险,NNT 相互关系举例;OR?RR ?HR;;不确定原则;Uncertainty Principle;;不确定原则的依据;LDL LOWERING AND IVUS (OVER 6M POST ACS);A to Z: COMPOSITE PRIMARY END-POINT;(标示值,